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First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia.

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Title: First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia.
Authors: Djaksigulova D; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Kaad SG; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Korecki AJ; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Lam SL; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Lengyell TC; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Simpson EM; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada. simpson@cmmt.ubc.ca.; Department of Medical Genetics, The University of British Columbia, Vancouver, BC, Canada. simpson@cmmt.ubc.ca.
Source: Gene therapy [Gene Ther] 2026 Mar 09. Date of Electronic Publication: 2026 Mar 09.
Publication Type: Journal Article
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1476-5462
DOI:10.1038/s41434-026-00605-5